Search / Trial NCT06611436

BeCoMe-9: a Clinical Study of BE-101 for the Treatment of Adults with Moderately Severe or Severe Hemophilia B

Launched by BE BIOPHARMA · Sep 20, 2024

Trial Information

Current as of December 21, 2024

Recruiting

Keywords

Hemophilia B Dose Escalation

ClinConnect Summary

The BeCoMe-9 clinical trial is studying a new treatment called BE-101 for adults with moderately severe or severe Hemophilia B, a condition where the blood doesn’t clot properly due to a lack of a protein called Factor IX (FIX). This trial is the first time BE-101 is being tested in humans. The treatment involves a single intravenous (IV) dose that aims to help the body produce enough FIX to improve blood clotting and reduce bleeding episodes. BE-101 is made using the patient’s own cells and a special gene-editing technique called CRISPR to insert the FIX gene into those cells.

To participate in this study, men aged 18 and older with moderately severe to severe Hemophilia B who have had prior treatment with Factor IX products are eligible. They should also be currently receiving preventative treatment and have good organ function. Participants will undergo a procedure to collect their own cells before receiving the treatment. Throughout the trial, they will be closely monitored for safety and how well the treatment works. Please note that there are specific health conditions that may exclude someone from participating, so it’s important to discuss eligibility with a healthcare provider.

Gender

MALE

Eligibility criteria

  • Inclusion Criteria:
  • Adult Males (≥18) with moderately severe to severe Hemophilia B (FIX deficiency)
  • Received ≥50 exposure days to Factor IX products preceding enrollment.
  • Currently receiving prophylaxis treatment
  • Adequate organ function and clinical labs
  • Able to tolerate study procedures including leukapheresis.
  • Exclusion Criteria:
  • Pre-existing or history of specific diseases
  • B-Cell malignancy, EBV lymphoproliferative disease
  • Primary immunodeficiency disease or disorder (PIDD) or systemic immuno-suppression
  • Arterial and/or venous thromboembolic events within 2 years prior to dosing
  • History of anaphylaxis or nephrotic syndrome
  • Active infection (HIV, Hep B or C)
  • History of inhibitor to FIX or inhibitor
  • History of an allergic reaction or anaphylaxis to FIX products
  • * Within 6 months from BE-101 administration:
  • Planned surgical procedure
  • Previously dosed with gene therapy or participated in an interventional clinical study
  • Planned participation in clinical trial within one year after BE-101
  • Administration of a vaccine within 28 days of dosing
  • Other protocol-defined inclusion/exclusion criteria may apply.

About Be Biopharma

Be Biopharma is a pioneering biotechnology company dedicated to advancing innovative therapies for patients with unmet medical needs. With a focus on harnessing the power of biologics and cutting-edge research, Be Biopharma is committed to developing next-generation treatments that improve patient outcomes across various therapeutic areas. The company emphasizes collaboration, scientific excellence, and a patient-centric approach, driving progress in clinical trials and ensuring the highest standards of safety and efficacy in its product development pipeline. Through its dedication to research and development, Be Biopharma aims to transform the landscape of healthcare and enhance the quality of life for individuals worldwide.

Locations

Minneapolis, Minnesota, United States

Ann Arbor, Michigan, United States

People applied

Timeline

First submit

Trial launched

Trial updated

Estimated completion

Not reported

Discussion 0